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Toward Patient-Centered Innovation: A Conceptual Framework for Patient-Reported Outcome Measures for Transformative Kidney Replacement Devices.

ABSTRACT: Individuals with dialysis-dependent kidney failure experience considerable disease- and treatment-related decline in functional status and overall well-being. Despite these experiences, there have been few substantive technological advances in KRT in decades. As such, new federal initiatives seek to accelerate innovation. Historically, integration of patient perspectives into KRT product development has been limited. However, the US Food and Drug Administration recognizes the importance of incorporating patient perspectives into the total product life cycle (i.e., from product conception to postmarket surveillance) and encourages the consideration of patient-reported outcomes in regulatory-focused clinical trials when appropriate. Recognizing the significance of identifying patient-reported outcome measures (PROMs) that capture contemporary patient priorities, the Kidney Health Initiative, a public-private partnership between the American Society of Nephrology and US Food and Drug Administration, convened a workgroup to (1) develop a conceptual framework for a health-related quality of life PROM; (2) identify and map existing PROMs to the conceptual framework, prioritizing them on the basis of their supporting evidence for use in the regulatory environment; and (3) describe next steps for identifying PROMs for use in regulatory clinical trials of transformative KRT devices. This paper summarizes the proposed health-related quality-of-life PROM conceptual framework, maps and prioritizes PROMs, and identifies gaps and future needs to advance the development of rigorous, meaningful PROMS for use in clinical trials of transformative KRT devices.

SUBMITTER: Flythe JE

PROVIDER: S-EPMC7536748 | biostudies-literature | 2020 Oct

REPOSITORIES: biostudies-literature

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Toward Patient-Centered Innovation: A Conceptual Framework for Patient-Reported Outcome Measures for Transformative Kidney Replacement Devices.

Flythe Jennifer E JE Hilliard Tandrea S TS Ikeler Kourtney K Keller San S Gipson Debbie S DS Grandinetti Amanda C AC Nordyke Robert J RJ Perrone Ronald D RD Roy-Chaudhury Prabir P Unruh Mark M West Melissa M Bocell Fraser F Hurst Frank P FP

Clinical journal of the American Society of Nephrology : CJASN 20200410 10

Individuals with dialysis-dependent kidney failure experience considerable disease- and treatment-related decline in functional status and overall well-being. Despite these experiences, there have been few substantive technological advances in KRT in decades. As such, new federal initiatives seek to accelerate innovation. Historically, integration of patient perspectives into KRT product development has been limited. However, the US Food and Drug Administration recognizes the importance of incor ...[more]

PMID: 32276947

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Project description:BackgroundPatient-reported outcome measures (PROMs) assess disability and progress toward functional goals while promoting patient-centered practice. They can be used by health professionals in any specialty and in a multitude of settings. This study reviews implementation strategies and lessons learned in a capacity-building program that took place with pediatric physiotherapists in Rwanda.MethodsUse of PROMs and patient-centered practice were integrated into 4 consecutive continuing professional development courses offered to 164 participants in Rwanda. We sought to identify a simple generic measure with proven validity in cross-cultural settings. The Patient-Specific Functional Scale was chosen due to its ease of use and ability to measure change in a wide range of patient conditions. Didactic classroom training and clinical site visits were 2 essential pedagogical elements of the capacity-building strategy. Site visits allowed for evaluation of skill levels and facilitation of knowledge transfer to patient care settings. Unique pairs of Rwandan colleagues were trained to serve as coteachers in each course to maximize sustainability of new techniques. This study presents data on a subset of 65 participants who completed a 48-hour pediatric rehabilitation course.ResultsAfter classroom instruction, 78% of participants were observed independently determining functional limitations with their patients. Additionally, pre- and post-tests indicated that therapists substantially increased their understanding of patient-centered practice after attending courses. Interviews conducted 26 months after the conclusion of the project revealed mixed success in sustainability of the use of PROMs, although perceived confidence remained high.ConclusionChallenges in long-term sustainability of new practices call attention to the need to target not only clinicians when introducing new methodologies, but also the Ministry of Health, hospital administration, and university faculty. Lessons learned from this study may be useful to other medical professionals planning capacity-building programs in low- and middle-income countries.

Project description:Background and aimsPatient-reported outcome measures (PROMs) for children (aged ≤ 18 years) present methodological challenges. PROMs can be categorised by their diverse underlying conceptual bases, including functional, disability and health (FDH) status; quality of life (QoL); and health-related quality of life (HRQoL). Some PROMs are designed to be accompanied by preference weights. PROMs should account for childhood developmental differences by incorporating age-appropriate health/QoL domains, guidance on respondent type(s) and design. This systematic review aims to identify generic multidimensional childhood PROMs and synthesise their characteristics by conceptual basis, target age, measurement considerations, and the preference-based value sets that accompany them.MethodsThe study protocol was registered in the Prospective Register of Systematic Reviews (CRD42021230833), and reporting followed Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. We conducted systematic database searches for generic multidimensional childhood PROMs covering the period 2012-2020, which we combined with published PROMs identified by an earlier systematic review that covered the period 1992-2011. A second systematic database search identified preference-based value sets for generic multidimensional PROMs. The PROMs were categorised by conceptual basis (FDH status, QoL and HRQoL) and by target age (namely infants and pre-schoolers aged < 5 years, pre-adolescents aged 5-11, adolescents aged 12-18 and multi-age group coverage). Descriptive statistics assessed how PROM characteristics (domain coverage, respondent type and design) varied by conceptual basis and age categories. Involvement of children in PROM development and testing was assessed to understand content validity. Characteristics of value sets available for the childhood generic multidimensional PROMs were identified and compared.ResultsWe identified 89 PROMs, including 110 versions: 52 FDH, 29 QoL, 12 HRQoL, nine QoL-FDH and eight HRQoL-FDH measures; 20 targeted infants and pre-schoolers, 29 pre-adolescents, 24 adolescents and 37 for multiple age groups. Domain coverage demonstrated development trajectories from observable FDH aspects in infancy through to personal independence and relationships during adolescence. PROMs targeting younger children relied more on informant report, were shorter and had fewer ordinal scale points. One-third of PROMs were developed following qualitative research or surveys with children or parents for concept elicitation. There were 21 preference-based value sets developed by 19 studies of ten generic multidimensional childhood PROMs: seven were based on adolescents' stated preferences, seven were from adults from the perspective of or on behalf of the child, and seven were from adults adopting an adult's perspective. Diverse preference elicitation methods were used to elicit values. Practices with respect to anchoring values on the utility scale also varied considerably. The range and distribution of values reflect these differences, resulting in value sets with notably different properties.ConclusionIdentification and categorisation of generic multidimensional childhood PROMs and value sets by this review can aid the development, selection and interpretation of appropriate measures for clinical and population research and cost-effectiveness-based decision-making.

Project description:BackgroundKnee replacement is a very effective and indispensable treatment option for end-stage knee arthritis, and the number of cases has been increasing worldwide. A replaced knee joint without patient joint awareness is thought to be the ultimate goal of artificial knees. Joint awareness reportedly correlates with patient satisfaction. Although numbness around a replaced knee is a minor but common problem, its effect on postoperative outcome is controversial. Joint awareness also is sensitive to subtle abnormalities of the joint, so it must be negatively affected by numbness. Although numbness is minor, it cannot be ignored to further improve knee replacement outcomes. This study investigated the relationship between patient-reported numbness and other patient-reported outcome measures (PROMs), including joint awareness, and kneeling. We developed a numbness score based on a 5-point Likert scale on frequency of numbness, with an intraclass correlation coefficient of 0.76 and higher scores indicating less numbness.MethodsThe numbness score, New Knee Society Score (KSS), Knee Injury and Osteoarthritis Outcome Score (KOOS), Forgotten Joint Score-12 (FJS-12), and other clinical and radiological data from 311 patients (394 primary knee replacements) were analyzed. Kneeling ability was evaluated by using kneeling-specific items in the KSS (KSS-Kneeling).ResultsNo numbness was found in 170 knees (43.1%), and some degree of numbness was found in the remaining 224 knees (56.9%). The numbness score showed weak-to-moderate correlations with KSS-Symptoms (r = 0.44), KSS-Satisfaction (r = 0.41), KSS-Activities (r = 0.29), and all KOOS subscales (r = 0.23-0.44), and FJS-12 (r = 0.42). Multiple regression analyses suggested that midline incision positively affected the numbness score over the anteromedial incision (p = 0.04) and that a better numbness score (p = 0.001), male sex (p < 0.0001), and better postoperative knee flexion angle (0.04) positively affected kneeling.ConclusionsThe numbness score positively correlated with PROMs and positively affected kneeling. Knee replacements performed via an anteromedial incision may be at higher risk for numbness.

Project description:BackgroundLiving with type 1 diabetes is challenging, and to support self-management, repeated consultations in specialist outpatient care are often required. The emergence of new digital solutions has revolutionized how health care services can be patient centered, providing unprecedented opportunities for flexible, high-quality care. However, there is a lack of studies exploring how the use of digital patient-reported outcome measures (PROMs) for flexible specialist care affects diabetes self-management. To provide new knowledge on the relevance of using PROMs in standard care, we have designed a multimethod prospective study.ObjectiveThe overall aim of this protocol is to describe our prospective multimethod observational study designed to investigate digital PROMs in a routine specialist outpatient setting for flexible patient-centered diabetes care (DigiDiaS).MethodsThis protocol outlines the design of a multimethod prospective observational cohort study that includes data from electronic health records, self-reported questionnaires, clinical consultation field observations, and individual in-depth interviews with patients and diabetes health care personnel. All patients with type 1 diabetes at a designated outpatient clinic were invited to participate and use the digital PROM implemented in clinical care. Both users and nonusers of the digital PROM were eligible for the prospective study, allowing for a comparison of the two groups. Data were collected at baseline and after 12 months, including self-management as the primary outcome assessed using the Patient Activation Measure, along with the secondary outcomes of digital health literacy, quality of life, health economy, and clinical variables such as glycated hemoglobin.ResultsThe digital solution was implemented for routine clinical care in the department in November 2021, and data collection for the prospective study started in October 2022. As of September 6, 2023, 84.6% (186/220) of patients among those in the digital PROM and 15.5% (34/220) of patients among the nonusers have consented to participate. We expect the study to have enough participants by the autumn of 2023. With 1 year of follow-up, the results are expected by spring 2025.ConclusionsIn conclusion, a multimethod prospective observational cohort study can offer valuable insights into the relevance, effectiveness, and acceptability of digital tools using PROMs in diabetes specialist care. Such knowledge is crucial for achieving broad and successful implementation and use of these tools in a large diabetes outpatient clinic.International registered report identifier (irrid)DERR1-10.2196/52766.

Project description:BackgroundCompetition-promoting reforms and economic incentives are increasingly being introduced worldwide to improve the performance of healthcare delivery. This study considers such a reform which was initiated in 2009 for elective hip replacement surgery in Stockholm, Sweden. The reform involved patient choice of provider, free establishment of new providers and a bundled payment model. The study aimed to examine its effects on hip replacement surgery quality as captured by patient reported outcome measures (PROMs) of health gain (as indicated by the EQ-5D index and a visual analogue scale (VAS)), pain reduction (VAS) and patient satisfaction (VAS) one and six years after the surgery.MethodsUsing patient-level data collected from multiple national registers, we applied a quasi-experimental research design. Data were collected for elective primary total hip replacements that were carried out between 2008 and 2012, and contain information on patient demography, the surgery and PROMs at baseline and at one- and six-years follow-up. In total, 36,627 observations were included in the analysis. First, entropy balancing was applied in order to reduce differences in observable characteristics between treatment groups. Second, difference-in-difference analyses were conducted to eliminate unobserved time-invariant differences between treatment groups and to estimate the causal treatment effects.ResultsThe entropy balancing was successful in creating balance in all covariates between treatment groups. No significant effects of the reform were found on any of the included PROMs at one- and six-years follow-up. The sensitivity analyses showed that the results were robust.ConclusionsCompetition and bundled payment had no effects on the quality of hip replacement surgery as captured by post-surgery PROMs of health gain, pain reduction and patient satisfaction. The study provides important insights to the limited knowledge on the effects of competition and economic incentives on PROMs.

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Toward Patient-Centered Innovation: A Conceptual Framework for Patient-Reported Outcome Measures for Transformative Kidney Replacement Devices.

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Toward Patient-Centered Innovation: A Conceptual Framework for Patient-Reported Outcome Measures for Transformative Kidney Replacement Devices.

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